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Experimental Drug May Protect From Type 1 Diabetes

JDRF-funded researchers at Harvard School of Public Health have found that an experimental drug, called TUDCA, can dramatically reduce the occurrence of type 1 diabetes (T1D) in mice specially bred to develop the disease. Presented at the American Diabetes Association’s (ADA) 72nd Scientific Sessions in Philadelphia, they reported that TUDCA protects beta cells from a stress response that occurs in beta cells under diabetic or pre-diabetic conditions. The primary function of the pancreatic beta cell is to synthesize and secrete insulin in response to glucose. Under normal conditions, insulin is produced by the beta cell in a highly controlled manner. But excess glucose or other stresses in the body can alter the ability of the beta cell to secrete sufficient insulin, and beta cells may become overwhelmed and die. This pathological stress response is increasingly thought to be a contributing factor in T1D as well as type 2 diabetes. TUDCA is currently in clinical study as a potential treatment for people with signs or symptoms of type 2 diabetes.

“Based on earlier work, we expected this might be a useful approach for reducing the incidence of type 1 diabetes, but we were surprised by the magnitude of the effect,” said Gokhan Hotamisligil, Ph.D. the principal investigator of the study. “Given that this mechanism of beta cell destruction and early onset diabetes is shared between experimental rodent models and humans, the potential for translating these findings to humans is clear. While TUDCA may not be the optimal therapy due to its chemical properties, these findings hold great promise for guiding the development of an improved therapy for the prevention or treatment of recent onset type 1 diabetes.”

In the work presented at the ADA meeting by Dr. Hotamisligil’s post-doctoral fellow, Feyza Engin, Ph.D., mice bred to get T1D were treated with TUDCA for four weeks. At the end of the treatment period, the treated mice experienced significantly lower inflammation in the pancreas compared to untreated mice. In another experiment, T1D-prone mice were treated daily with TUDCA for 30 weeks and blood glucose levels were monitored daily to detect the onset of T1D. As a result of this treatment, only 10 percent of the TUDCA-treated mice developed T1D compared to almost half of the untreated mice. Dr. Engin also went on to show that biochemical changes in the stress response in beta cells from the animals correlate with similar changes in isolated human beta cells obtained from people with T1D.